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Efgartigimod as a treatment for adults with primary immune thrombocytopenia: a plain language summary of the ADVANCE
Catherine Broome1, Yoshitaka Miyakawa2, Monica Carpenedo3
1Lombardi Cancer Center, Division of Hematology, Georgetown University, Washington, DC, USA.
What is this summary about? This is a plain language summary of an article published in the scientific journal The Lancet in 2023. The ADVANCE IV study involved people living with immune thrombocytopenia, called ITP. In primary ITP your immune system attacks platelets in the blood. People with ITP have a lower number of platelets than normal. Some people may bruise or bleed more easily, and bleeding can become serious if platelet counts are very low. ITP is called persistent when low platelet counts continue for 3 to 12 months and chronic if low platelet counts continue for more than a year from diagnosis. What happened in this study? Participants received efgartigimod or a placebo (inactive medicine) through a needle into the vein (intravenous) weekly. The main aim was to see how many people with chronic ITP had increased platelet counts over several weeks during the treatment period. Researchers looked at how well intravenous efgartigimod worked in improving symptoms such as bleeding in adults with chronic or persistent primary ITP. They also looked at the side effects people had during the study. What were the results of this study? Overall, 131 people with ITP participated in the study between December 2019 and February 2022. On average, people had received their ITP diagnosis over 10 years before the start of the study. Around 7 in 10 people (67%) had previously received at least 3 other treatments for ITP. More people (22%) who received intravenous efgartigimod had increased platelet counts than those who received a placebo (5%). Over half of the people who received efgartigimod had improvements when taking platelet counts and bleeding into account. The most common side effects were tiny purple, red, or brown spots on the skin (petechiae), red blood cells in the urine, and headaches. These were seen at similar rates in people receiving efgartigimod and those receiving a placebo. What do the results mean? These results show that intravenous efgartigimod helps increase platelet counts in people with chronic or persistent primary ITP who had tried several previous treatments for ITP.
What is this summary about? This is a plain language summary of an article published in the scientific journal The Lancet in 2023. The ADVANCE IV study involved people living with immune thrombocytopenia, called ITP. In primary ITP your immune system attacks platelets in the blood. People with ITP have a lower number of platelets than normal. Some people may bruise or bleed more easily, and bleeding can become serious if platelet counts are very low. ITP is called persistent when low platelet counts continue for 3 to 12 months and chronic if low platelet counts continue for more than a year from diagnosis. What happened in this study? Participants received efgartigimod or a placebo (inactive medicine) through a needle into the vein (intravenous) weekly. The main aim was to see how many people with chronic ITP had increased platelet counts over several weeks during the treatment period. Researchers looked at how well intravenous efgartigimod worked in improving symptoms such as bleeding in adults with chronic or persistent primary ITP. They also looked at the side effects people had during the study. What were the results of this study? Overall, 131 people with ITP participated in the study between December 2019 and February 2022. On average, people had received their ITP diagnosis over 10 years before the start of the study. Around 7 in 10 people (67%) had previously received at least 3 other treatments for ITP. More people (22%) who received intravenous efgartigimod had increased platelet counts than those who received a placebo (5%). Over half of the people who received efgartigimod had improvements when taking platelet counts and bleeding into account. The most common side effects were tiny purple, red, or brown spots on the skin (petechiae), red blood cells in the urine, and headaches. These were seen at similar rates in people receiving efgartigimod and those receiving a placebo. What do the results mean? These results show that intravenous efgartigimod helps increase platelet counts in people with chronic or persistent primary ITP who had tried several previous treatments for ITP.
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